IBRX apparantly received encouragement in January that the unmet need in BCG unresponsive Papillary desease outweigh the requirement for a placebo controlled trial.
But the FDA position on single arm trials seems to be changing with the termination of Marks., Dr. Rachel Sherman (who it is said "pioneered single arm trials") and Kennedy's ideas about more rigorous testing.
Prasad’s appointment (https://www.biospace.com/fda/vinay-prasads-fda-appointment-anything-but-status-quo) confirms this; and, I think the RTF letter reflects a reluctance of the current FDA to move forward without more evidence the drug is effective and safe in BCG unresponsive Papillary cancer, especially as new competitive drugs are in the pipeline.
It is my understanding that cases of Papillary disease both outnumber CIS, but also are slower progressing and less dangerous and the need for accelerated approval may be less important.
The existing approval of Anktiva was based on QUILT-3.032 Cohort A, involving CIS, a multifocal cancer that is more agreessive than Papillary, even the high grade Papillary tested alone in Cohart B.
I predict the FDA is going to require a confirmatory trial with placebos, but we have yet to learn whether they will accept the sNBA subject to that trial confirmation coming AFTER accelerated approval.
IMO, PSS is going to seek try to get a conditiional approval in the Type A meeting, subject to a confirmatory randomized placebo controlled trial.
If the FDA refuses that, IBRX probably will be required to do a 6 month to 1 year trial of Papillary with placebos to satisfy the efficacy requirement. I don't think they will seek an advisory committee opinion, but that is another possibility.
As usual, I disclose that I am relying on Chat-GPT research, not personal expertise, so take my views with a grain of salt.. |
